Recently Approved Treatment for Dravet Syndrome Continues to Show Impacts in Phase 3 Trial
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Recently Approved Treatment for Dravet Syndrome Continues to Show Impacts in Phase 3 Trial

According to a story from BioSpace, the biopharmaceutical company Zogenix, Inc., has recently announced the release of results from a phase 3 clinical trial testing the company's drug fenfluramine (marketed…

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AI System Created To Speed Up Epileptic Diagnosis is Tested on Dravet Syndrome
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AI System Created To Speed Up Epileptic Diagnosis is Tested on Dravet Syndrome

As reported in Dravet Syndrome News, in a new study, researchers created an artificial intelligence system which could diagnose epileptic conditions more rapidly and easily than existing systems. Epilepsy is…

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This Mother was Prepared to Break the Law to Treat her Daughter’s Dravet Syndrome
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This Mother was Prepared to Break the Law to Treat her Daughter’s Dravet Syndrome

According to a story from Mirror Online, Vera Twomey from Cork, Ireland, was devastated when her four month old daughter had a severe seizure that lasted 45 minutes. Before long,…

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ICYMI: Positive Phase 3 Trial Results for Lennox-Gastaut Syndrome
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ICYMI: Positive Phase 3 Trial Results for Lennox-Gastaut Syndrome

Lennox-Gastaut syndrome (LGS) is a form of epilepsy.  Most patients with this condition are resistant to current treatments and experience seizures throughout the duration of their life. They may face…

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With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two…

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These Two Companies Just Announced a Partnership to Develop Multiple Gene Therapies
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These Two Companies Just Announced a Partnership to Develop Multiple Gene Therapies

According to a story from BioSpace, the gene therapy companies Sarepta Therapeutics, Inc. and StrideBio, Inc. have recently announced the completion of a licensing and collaboration agreement that could lead…

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CBD-Based Treatment for Rare Childhood Epilepsies Gets Recommendation from NICE
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CBD-Based Treatment for Rare Childhood Epilepsies Gets Recommendation from NICE

According to a story from BioPortfolio, the biopharmaceutical company GW Pharmaceuticals plc recently announced that its medicine Epidyolex has earned recommendation from the UK National Institute for Health and Care…

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Initial Trial Data Looks Promising for a Potential Treatment for Rare Epilepsies
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Initial Trial Data Looks Promising for a Potential Treatment for Rare Epilepsies

According to a story from sectorpublishingintelligence.co.uk, the drug company Ovid Therapeutics Inc. has recently announced that the early data from its phase 2 clinical trial is mostly encouraging. This clinical…

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Experimental Treatment for Dravet Syndrome Earns Orphan Drug Designation
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Experimental Treatment for Dravet Syndrome Earns Orphan Drug Designation

According to a story from Pharmaceutical Technology, the company Stoke Therapeutics experimental drug candidate, known as STK-001, has earned Orphan Drug designation from the US Food and Drug Administration (FDA).…

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A Nasal Spray Has Just Become the First New Therapy Approved by the FDA for Cluster Seizures in 20 Years

Cluster Seizures Approximately 1/3 of all epilepsy patients live with uncontrolled seizures. Of these, around 150,000 in the United States alone  experience cluster seizures (otherwise known as acute-repetitive seizures, crescendo…

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Scotland Only Allows 10 Children to Receive New Cannabis-Based Seizure Drug
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Scotland Only Allows 10 Children to Receive New Cannabis-Based Seizure Drug

  There are over 500 children in Scotland whose parents feel could benefit from a new cannabis-derived drug for seizures called Epidiolex. But recently, Secretary Jeanne Freeman had to tell…

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A Recap on 2018: Record Number of Drugs Approved by the FDA for Rare Diseases

Each year the Food and Drug Administration approves dozens of new therapies to fulfill unmet needs for various patient populations. It's just been published that in 2018 they approved a…

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Potential Dravet Syndrome Treatment Shows Capability in Clinical Trial
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Potential Dravet Syndrome Treatment Shows Capability in Clinical Trial

According to a story from pm360online.com, the pharmaceutical company Zogenix Inc. recently announced that its experimental drug candidate ZX008, also known as Fintepla®, has performed well in the company's latest…

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The FDA is Modernising Their Drug Development Processes
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The FDA is Modernising Their Drug Development Processes

In a recent statement, Scott Gottlieb, the Commissioner of the US Food and Drug Administration, has re-affirmed the organisation’s commitment to modernising drug regulatory pathways. New changes are hoped to…

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